ASCO Roundup: Checkpoints, Pushback Against Prices, CAR-T & More

a San Francisco Bay Area R&D power—hinge upon two events: potential FDA approval on August 11 of its cobimetinib, which is controlled by Genentech, and Phase 3 results in renal cell carcinoma for its drug cabozantinib (Cometriq)

—An experimental brain cancer vaccine from Hampton, NJ, and Needham, MA-based Celldex Therapeutics (NASDAQ: [[ticker:CLDX]]) continued to show promise, helping extend the lives of a small group of patients whose recurrent glioblastoma—an aggressive, deadly brain cancer—carries a specific genetic mutation.

These are patients who typically live only a handful of months and have few options left, which is why Celldex wants to use the study to build its case for an accelerated approval, as the study’s lead investigator told Xconomy.

—Waltham, MA-based ImmunoGen (NASDAQ: [[ticker:IMGN]]) was a surprise Wall Street winner at ASCO. Shares of the company skyrocketed 72 percent after ImmunoGen said that in a small study, 9 of 17 patients with platinum-resistant ovarian cancer responded to its experimental antibody-drug conjugate, mivretuximab soravtansine. Six of those nine patients were still responding after data cutoff, with five of those on drug for over 15 weeks. While this is from a Phase 1 study and the sample size is small, RBC Capital Markets analyst Simos Simeonidis noted the response rate is “well above” the typical 15 to 20 percent seen from such patients. ImmunoGen will begin a mid-stage study later this year that “could be used for registration,” executives said in a statement.

—There were advances in the search for new treatments for the deadly blood cancer multiple myeloma. A few weeks ago, Bristol and AbbVie shared Phase 3 data from the antibody-based drug, elotuzumab, which helped slow the progression of the disease in a Phase 3 study when combined with the standard of care—dexamethasone and Celgene’s lenalidomide (Revlimid). This weekend, Johnson & Johnson said an antibody it licensed from Denmark’s Genmab, daratumumab, helped extend the lives of a group of very sick patients who had stopped responding to other therapies. Here’s more on daratumumab from Reuters and Targeted Oncology.

—Lost in all the ASCO immunotherapy news was Cambridge, MA-based Dicerna Pharmaceuticals’s (NASDAQ: [[ticker:DRNA]]) first look at human clinical data. Dicerna is developing an RNA interference therapy meant to target the “undruggable” Myc gene, which is mutated in several forms of cancer.

Dicerna said it has seen anti-tumor activity in two of three patients with advanced pancreatic neuroendocrine tumors (PNET) who have failed prior therapies. These data are part of an ongoing Phase 1 study of patients with a variety of cancers. Given the early results, Dicerna is adding an arm to the trial specifically for more PNET patients. This was only a minor update from Dicerna’s ASCO abstract in mid-May, however; a more substantial update on Dicerna’s RNAi efforts in cancer is expected later in the year.

Ben Fidler contributed to this report.

Photo of Chicago skyline courtesy Tony Webster via a Creative Commons license.

Author: Alex Lash

I've spent nearly all my working life as a journalist. I covered the rise and fall of the dot-com era in the second half of the 1990s, then switched to life sciences in the new millennium. I've written about the strategy, financing and scientific breakthroughs of biotech for The Deal, Elsevier's Start-Up, In Vivo and The Pink Sheet, and Xconomy.